Cambridge Healthtech Institute’s Inaugural
Cell Therapy CMC, Quality and Analytics
Regulatory, Analytical and Quality Considerations for Cell Therapies
Part of CHI's 8th Annual The Bioprocessing Summit

August 15-16, 2016 | Westin Boston Waterfront | Boston, MA

CHI’s Cell Therapy CMC, Quality and Analytics meeting is the only conference that takes an in-depth, practical, case study driven approach to the analysis, regulation and quality control of cell-based and gene-modified cell therapies, with particular attention paid to defining ancillary materials, ensuring product consistency, setting CQAs and CPPs, CMC strategies, potency assay development, method validation, comparability studies, stability and setting specifications.

Final Agenda

Monday, August 15

8:00 am Short Course Registration

9:00 – 11:30 Recommended Morning Short Course*
SC4: Advanced Process Control Strategies in Bioprocessing

* Separate registration required

11:30 Main Conference Registration Opens


1:00 pm Chairperson’s Opening Remarks

Christopher Bravery, Ph.D., Consultant Regulatory Scientist, Consulting on Advanced Biologicals Ltd.

Classification of Cell Therapies and Recent Trends

Alexey_BersenevAlexey Bersenev, Ph.D., Director, Advanced Cell Therapy Lab, Yale University

Cell therapy is administration of living cells in human with therapeutic purpose. In this presentation I will discuss classification and categories of cell therapies in terms of cell types, therapeutic purpose, degree of manipulation and regulatory pathways. I will highlight differences between immunocellular therapies and regenerative medicine and present recent trends in academic and commercial development of advanced cell therapies.

1:45 Defining and Qualifying Raw Materials for Use in Cell Therapies

Fouad_AtoufFouad Atouf, Ph.D., Director, United States Pharmacopeia

Qualification of raw materials used in the manufacturing of cellular therapies, require the use of risk assessment strategies to categorize the critical components of a manufacturing process. In addition to cell culture supplements, excipients and other formulation’s components must meet the required quality to ensure consistency in manufacturing and subsequently the quality and safety of finished cell therapy products. Testing strategies for raw materials may be achieved by the use of established and validated procedures as well as the availability of reference materials.

2:15 Ensuring Quality of Cellular Starting Material

Elizabeth_ReadElizabeth Read, M.D., Senior Vice President, Product Development, Medeor Therapeutics, Inc.

For cell therapy products, the starting material contains living, functional cells that become an integral part of the active drug substance and final product. Quality specifications for the donor, collection process, and shipping/storage/hold steps of starting material should be considered early in CMC development. Donor-to-donor variability, availability of clinically representative starting materials, and starting material stability create unique development challenges.

2:45 Refreshment Break


3:15 Developing a Clinical Grade Process Starting from a Research Method: Practical Consideration of Raw Materials for Cell Therapy Production

Benjamin_FryerBenjamin Fryer, Ph.D., Team Leader, Processing/Manufacturing, Heart Regeneration Program, University of Washington School of Medicine

In order to generate an approved cell therapy an aseptic and well controlled manufacturing process must be developed. This begins with proper raw material sourcing via secure and trust worthy supply chains, and material validation via identity and/or in-process testing. Planning ahead in early stage development should facilitate development of a scalable cold supply chain cell therapy product.

3:45 Sponsored Presentation (Opportunity Available)

4:00 Panel Discussion: Navigating International Regulations for Cell and Gene Therapies

Christopher_BraveryChristopher Bravery, Ph.D., Consultant Regulatory Scientist, Consulting on Advanced Biologicals Ltd.

Scott_BurgerScott Burger, M.D., Consultant, Advanced Cell and Gene Therapy

  • Global Regulatory Updates and Developments
  • Building a Regulatory Strategy
  • Common Regulatory Issues

4:30 Breakout Discussions

This session provides an opportunity to discuss a focused topic with peers from around the world in an open, collegial setting. Select from the list of topics available and join the moderated discussion to share ideas, gain insights, establish collaborations, or commiserate about persistent challenges.

It's the CMC, Stupid

Dr Anthony Davies, Ph.D., President, Dark Horse Consulting

Potency Assay Design and Validation

Christopher Bravery, Ph.D., Consultant Regulatory Scientist, Consulting on Advanced Biologicals Ltd.

Scott Burger, M.D., Consultant, Advanced Cell and Gene Therapy

5:30 Grand Opening Reception in the Exhibit Hall with Poster Viewing

7:00 End of Day

Tuesday, August 16

7:30 am Registration Opens and Morning Coffee


7:55 Chairperson’s Remarks

Bernadette Keane, Ph.D., Consultant, CMC/Quality/Regulatory Compliance, Specializing in Gene and Cell Therapies

8:00 Overcoming Challenges in Developing Potency Assays for Heterogeneous Cell Products

Therese_ChoquetteTherese Choquette, Ph.D., Fellow, TRD Cell & Gene Therapy, Novartis Pharmaceuticals

The ideal potency assay reflects the mechanism of action (MoA), predicts clinical response, is easy to perform and shows low variability. However, for cell and gene therapy products, often containing heterogenic cell populations, this ideal is rarely achievable. Several challenges exists; including unknown MoA, bystander activations, unspecific responses, and patient-to-patient variations. This presentation illustrates some of our experiences of potency assay development for CART cells.

8:30 QbD “Thinking” in the Development of Cell Therapy Products

Bernadette_KeaneBernadette Keane, Ph.D., Consultant, CMC/Quality/Regulatory Compliance, Specializing in Gene and Cell Therapies

Cell therapies, rely heavily on their manufacturing processes to meet final-product quality attributes. In the development of Allocetra (apoptotic cells), a patient-specific product, QbD approach was introduced at an early stage. Building quality into this product with an understanding of the manufacturing process while taking into account the end user, the patient, enabled the development of a well-defined reproducible product.

9:00 Fluorescent Probe-based Platform for Mesenchymal Stem Cell Quality Control

Deepak_RaghothamanDeepak Raghothaman, PhD,. Project Scientist, Bioprocessing Technology Institute (BTI), A*STAR

9:30 Sponsored Presentation (Opportunity Available)

9:45 Coffee Break in the Exhibit Hall with Poster Viewing


10:30 Use of Target Product Profile During Development

Stafano_BailiaStefano Baila, Ph.D., Manager, Process Development, Celyad

The Target Product Profile serves as the foundation of the product and clinical development plan, with the final product properties and use in mind. This approach takes all aspects into account, including quality, cost of goods, scalability and clinical use and ensures alignment of all stakeholders during the development phases.


11:00 A Multiparametric Approach for Defining Product Quality

Damian_MarshallDamian Marshall, Ph.D., Head, Analytical Department, Cell and Gene Therapy Catapult

The manufacture of cell therapy products can take several weeks or even months and involve a range of complex processing steps. As a result it can be challenging to define what cell characteristics are most appropriate to measure in order to ensure product quality is being maintained. This presentation will demonstrate a novel approach for marker screening and analysis that can be used to develop a framework to track product quality and to define the best approach for in-process control monitoring.

11:30 Early Process Development of CAR-T Therapies

 Ryan Crisman, PhD., Associate Director, Head of Late Stage Process Development, Juno Therapeutics

World Courier Amerisource Bergen  12:00 pm Ensuring Security Within the Cell Therapy Supply Chain>

Tim Redmond, Director, Regional Sales, Sales, World Courier, Inc.

The cell therapy supply chain needs expertise, which takes into account many elements. In this presentation Tim Redmond will share some of the expertise that World Courier has garnered through the years in cell therapy logistics.
 • Learn how to plan ahead for the transport of these critical shipments
 • Understand regulatory requirements for cross border shipments
 • Gain knowledge on how to select the proper packaging for all phases of the supply chain – from patient collection through to patient dosing
 • Understanding the Chain of Custody, and the importance of knowing where your shipments are at all times
 • Discover the role of specialized logistics to ensure the personalized care of each patient’s samples and therapeutic agents

Millipore Sigma12:30 Luncheon Presentation: Go Beyond: How to Make Novel Therapies a Reality in Tomorrow’s Complex Biopharma Landscape

Murrell_JulieJulie Murrell, Ph.D., Head, Cell Therapy Bioprocessing, MilliporeSigma

A recent survey conducted by the Economist Intelligence Unit highlights the new products that the biopharma industry identifies as most disruptive to their growth strategies in the next five years. The findings raise a challenge to biopharma to go beyond barriers to bringing new products to market by presenting ways to overcome these hurdles and make novel therapies a reality.

1:15 Dessert Refreshment Break in the Exhibit Hall with Poster Viewing


1:55 Chairperson’s Remarks

Bernadette Keane, Ph.D., Consultant, CMC/Quality/Regulatory Compliance, Specializing in Gene and Cell Therapies

2:00 Identifying Stability-Indicating Parameters for Stability Studies and Beyond

Christopher_BraveryChristopher Bravery, Ph.D., Consultant Regulatory Scientist, Consulting on Advanced Biologicals Ltd.

This talk will address the need to identify stability-indicating parameters for the product and discuss how accelerated and/or forced degradation studies can be useful to achieve this. Considerations for the design and execution of a comprehensive stability program and the use of stability data and stability-indicating parameters beyond stability studies will also be discussed.

2:30 Release Testing Considerations for Different Cell Therapy Paradigms

Joseph_SondejJoseph Sondej, Senior Manager, QC Operations, Celgene Cellular Therapeutics

Using examples this presentation will layout considerations for testing fresh and frozen cell therapy products as part of release testing considerations for different cell therapy paradigms..

3:00 Mitigating Risk of Process Change: Strategies and Tactics for Effective Comparability

Scott_BurgerScott Burger, M.D., Consultant, Advanced Cell and Gene Therapy

Manufacturing process changes are inevitable - processes are scaled up, methods and technology are improved, raw materials are changed, operations are transferred to new manufacturing sites. Any change to the manufacturing process, however, risks altering the cell therapy product itself. This session will discuss methods, tactics, and requirements for prospectively managing process changes and demonstrating comparability.

3:30 Refreshment Break in the Exhibit Hall with Poster Viewing


4:15 CAR-T Manufacturing and CMC Strategies

Nina_KotsopoulouNina Kotsopoulou, D.Phil., Director, Process Development, Autolus

Autolus is a biopharmaceutical company focused on delivering CAR-T cell products to patients. This presentation will focus on the challenges of combining efficient and fast delivery of Phase I/II suitable processes and products, with ensuring timely delivery at Phase III (and with minimal comparability risk) of improved, robust processes suitable for commercialisation.

4:45 Autologous Gene Therapy Manufacturing Comparability: Meeting the Challenge through Process Understanding and Product Characterization

Jeffrey_CramMr Jeffrey Cram, PhD., Scientist II, Technical Operations, Cellular Process Development and Manufacturing, bluebird bio

Demonstrating comparability of ex vivo autologous gene therapy products and processes across multiple facilities in the US and EU may be required to meet capacity requirements. A comparability plan should therefore incorporate elements to support both BLA and MAA as early in the development lifecycle as possible. A summary of comparability approaches for ex vivo autologous gene therapy products will be presented..

5:15 End of Conference